Single Ascending Dose Study in Participants With LCA10

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The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of AGN-151587 (EDIT-101) administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in intron 26 of the CEP290 gene ("LCA10-IVS26").

This is an open-label, single ascending dose study of AGN-151587 (EDIT-101) in adult and pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Approximately 18 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of AGN-151587 in this study. AGN-151587 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.

Testing the subjects visual function by having the subject walk through obstacle courses. Courses will have different levels of difficulty depending on the light levels of the room and the contrast of the objects in the room.

Change from baseline in LogMAR measurement of BCVA [ Time Frame: 1 year ]

The test will evaluate visual acuity in ranges from light perception to normal vision.

Change from baseline in pupillary response [ Time Frame: 1 year ]

Measuring the change in pupil diameter in response to a light stimulus.

Change from baseline in dark adapted visual sensitivity using Full field light sensitivity threshold (FST) [ Time Frame: 1 year ]

Flashes of light of varying luminance are presented to the eye and the subject reports is the flash was seen.

Change from baseline in macula thickness [ Time Frame: 1 year ]

Change from baseline in contrast sensitivity [ Time Frame: 1 year ]

The Lea symbols chart will be used for subjects under age 6 and the Pelli-Robson chart for all other subjects. The images or letters on the charts are in decreasing contrast.

Change from baseline in macular sensitivity as measured by microperimetry [ Time Frame: 1 year ]

Visual field test measuring the amount of light perceived in specific parts of the macula.

Change from baseline in color vision score using the Farnsworth 15 score [ Time Frame: 1 year ]

The Farnsworth D15 tests for congenital and acquired color vision defects. Fifteen color discs will be arranged by the subject. Scoring is accomplished by recording the sequence selected by the patient on a copy of the score sheet. A patient with a color vision deficiency will arrange the color discs in a different order than a person with normal color vision.

Change from baseline in QOL score for Age <8 years using the Children's Visual Function Questionnaire [ Time Frame: 1 year ]

Change from baseline in QOL score for Age 8 to <18 years using the Impact of Vision Impairment for Children [ Time Frame: 1 year ]

Change from baseline in QOL score for Age >18 years if BCVA is worse than 1.0 logMAR in both eyes using the Impact of Vision Impairment for Very Low Vision [ Time Frame: 1 year ]

Change from baseline in QOL score for Age >18 years if BCVA is 1.0 logMAR or better in both eyes using the Impact of Vision Impairment [ Time Frame: 1 year ]

Change from baseline in visual field using kinetic perimetry [ Time Frame: 1 year ]

Kinetic perimetry looks as the visual field to identify regions of normal and abnormal sensitivity to light

Change from baseline in Patient Global Impressions of Change score [ Time Frame: 1 year ]

This QOL has 5 non-numeric choices for the subject to select how they believe their condition has changed.

Change from baseline in gaze tracking [ Time Frame: 1 year ]

Video clips of the eyes are used to measure eye position and stability over time.

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Layout table for eligibility information

Ages Eligible for Study:

3 Years and older (Child, Adult, Older Adult)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Male or female

At least 3 years of age at screening with CEP290-related retinal degeneration caused by a homozygous or compound heterozygous mutation involving c.2991+1655A>G in IVS26 of the CEP290 gene.

Allergan will share de-identified patient-level data and study-level data including protocols and clinical study reports for phase 2 - 4 trials completed after 2008 that are registered to ClinicalTrials.gov or EudraCT, have received regulatory approval in the United States and/or the European Union in a given indication and the primary manuscript from the trial has been published. To request access to the data, the researcher must sign a data use agreement and any shared data is to be used for non-commercial purposes. More information can be found on http://www.allerganclinicaltrials.com/.

Supporting Materials:

Study Protocol
Clinical Study Report (CSR)

Time Frame:

After having received regulatory approval in the United States and/or the European Union in a given indication and the primary manuscript from the trial has been published.

Access Criteria:

To request access to the data, the researcher must sign a data use agreement and any shared data is to be used for non-commercial purposes.